Antisense nucleic acids
First Claim
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1. An antisense oligomer consisting of a nucleotide sequence selected from the group consisting of SEQ ID NOs:
- 25, 30, 33, 79, and 80, or pharmaceutically acceptable salt or hydrate thereof,wherein the antisense oligomer is a morpholino oligomer, a peptide nucleic acid (PNA), or an oligonucleotide comprising at least one nucleotide having;
(i) a modified sugar moiety, wherein the 2′
-OH group of a ribose is replaced by any one selected from the group consisting of R, R′
OR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br and I (wherein R is an alkyl or an aryl and R′
is an alkylene), or(ii) a modified phosphate-binding region selected from the group consisting of a phosphorothioate bond, a phosphorodithioate bond, an alkylphosphonate bond, a phosphoramidate bond, and a boranophosphate bond.
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Abstract
The present invention provides an oligomer which allows exon 45 skipping in the human dystrophin gene.
11 Citations
19 Claims
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1. An antisense oligomer consisting of a nucleotide sequence selected from the group consisting of SEQ ID NOs:
- 25, 30, 33, 79, and 80, or pharmaceutically acceptable salt or hydrate thereof,
wherein the antisense oligomer is a morpholino oligomer, a peptide nucleic acid (PNA), or an oligonucleotide comprising at least one nucleotide having; (i) a modified sugar moiety, wherein the 2′
-OH group of a ribose is replaced by any one selected from the group consisting of R, R′
OR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br and I (wherein R is an alkyl or an aryl and R′
is an alkylene), or(ii) a modified phosphate-binding region selected from the group consisting of a phosphorothioate bond, a phosphorodithioate bond, an alkylphosphonate bond, a phosphoramidate bond, and a boranophosphate bond. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8)
- 25, 30, 33, 79, and 80, or pharmaceutically acceptable salt or hydrate thereof,
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9. A pharmaceutical composition for treatment of muscular dystrophy, which comprises an antisense oligomer consisting of any one nucleotide sequence selected from the group consisting of SEQ ID NOs:
- 25, 30, 33, 79, and 80, or pharmaceutically acceptable salt or hydrate thereof as an active ingredient.
- View Dependent Claims (10, 14, 15, 16, 17, 18, 19)
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11. A method for treating muscular dystrophy, comprising the step of administering a muscular dystrophy patient with an antisense oligomer consisting of a nucleotide sequence selected from the group consisting of SEQ ID NOs:
- 25, 30, 33, 79, and 80, or pharmaceutically acceptable salt or hydrate thereof.
- View Dependent Claims (12, 13)
Specification